SRPT Finishes Enrolment in Late-Stage Study for Rare Muscular Disorder

Core Insights - Sarepta Therapeutics has completed enrollment and dosing in the phase III EMERGENE study for its gene therapy SRP-9003 targeting limb-girdle muscular dystrophy (LGMD) Type 2E/R4 [1][2] - Positive data from the EMERGENE study is anticipated in the first half of 2025, with plans for regulatory filing for accelerated approval if results are favorable [2] - LGMD2E/R4 is an ultra-rare genetic disease with no current treatment options beyond symptom management, leading to significant mobility loss and early mortality [3] Company Developments - Sarepta has initiated a clinical study for another gene therapy candidate, SRP-9004, targeting LGMD type 2D, and plans to start a study for SRP-9005 for LGMD type 2C in Q1 2025 [4][5] - The studies are designed to support FDA approval under the accelerated pathway [5] Stock Performance - Year-to-date, Sarepta's shares have increased by 24.7%, contrasting with a 10.5% decline in the industry [8] Commercial Portfolio - Sarepta's commercial portfolio includes three approved RNA-based PMO therapies and the one-shot gene therapy Elevidys, which is the first of its kind for DMD in the U.S. [9][10] - Elevidys generated approximately $437 million in product sales in the first nine months of 2024, compared to over$200 million in the previous year [10] - The company’s therapies, including Exondys 51, Vyondys 53, and Amondys 45, have strong demand and are expected to address nearly a third of all DMD patients in the U.S. [11]